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    Summary
    EudraCT Number:2012-004892-37
    Sponsor's Protocol Code Number:NL41205.018.12
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2014-12-03
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedUK - MHRA
    A.2EudraCT number2012-004892-37
    A.3Full title of the trial
    N-Acetylcysteine in patients with Sickle Cell Disease. Reducing the incidence of daily life pain in patients with sickle cell disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    N-Acetylcysteine in patients with Sickle Cell Disease: Reducing the incidence of daily life pain in patients with sickle cell disease
    A.3.2Name or abbreviated title of the trial where available
    N-Acetylcysteine in patients with Sickle Cell Disease
    A.4.1Sponsor's protocol code numberNL41205.018.12
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberISRCTN28828586
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01849016
    A.5.4Other Identifiers
    Name:EudraCTNumber:2012-004892-37
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAcademic Medical Center, Department of Haematology, University of Amsterdam
    B.1.3.4CountryNetherlands
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportAcademic Medical Center
    B.4.2CountryNetherlands
    B.4.1Name of organisation providing supportStichting JANIVO
    B.4.2CountryNetherlands
    B.4.1Name of organisation providing supportFonds NutsOhra
    B.4.2CountryNetherlands
    B.4.1Name of organisation providing supportThe Netherlands Organisation for Health Research and Development
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAcademic Medical Center
    B.5.2Functional name of contact pointMarjolien Spiering
    B.5.3 Address:
    B.5.3.1Street AddressMeibergdreef 9
    B.5.3.2Town/ cityAmstredam
    B.5.3.3Post code1105AZ
    B.5.3.4CountryNetherlands
    B.5.4Telephone number00 31 205665785
    B.5.5Fax number00 31 206919743
    B.5.6E-mailm.spiering@amc.nl
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name N-AcetylCysteine
    D.2.1.1.2Name of the Marketing Authorisation holderPharmazell GmbH
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameN-AcetylCysteine
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNN-Acetylcysteine
    D.3.9.1CAS number 616-91-1
    D.3.9.3Other descriptive nameAcetylcysteine, NAC
    D.3.9.4EV Substance CodeAS2
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number600
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Sickle Cell Disease
    E.1.1.1Medical condition in easily understood language
    Sickle Cell Disease
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level LLT
    E.1.2Classification code 10002077
    E.1.2Term Anaemia sickle cell
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level LLT
    E.1.2Classification code 10040643
    E.1.2Term Sickle cell crisis
    E.1.2System Organ Class 100000004851
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Does administration of N-acetylcysteine (NAC) tablets twice daily compared with placebo tablets reduce the frequency and severity of sickle cell related pain in daily life as evaluated by using pain diaries filled by study patients?
    E.2.2Secondary objectives of the trial
    The effect of NAC study medication on:
    - the severity of sickle cell disease(SCD)related pain
    - the frequency and severity of SCD crises
    - the frequency and length of hospital admissions
    - the societal costs of SCD related pain
    - the quality of life of participants
    - the use of pain medication at home
    - blood markers for inflammation, haemolysis, coagulation, adhesion and oxidative stress.

    The study will also assess the tolerability/safety of NAC tablets.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Age 12 years or older
    - Sickle cell disease, either homozygous sickle cell disease (HbSS), HbSC sickle cell
    disease, HbS0 or HbS+ thalassemia: Genotype needs to be confirmed by laboratory tests (high performance liquid chromatography).
    - History of at least 1.0 painful crisis per year in the past 3 years: A crisis will be defined here as a patient defined, painful, sickle cell related episode of at
    least 24 hours where a subject experienced significant impediments in his/her daily
    activities, and pain medication had to be taken. A visit to a medical facility is not obligatory in this definition.
    - Written informed consent from patient/parent/guardian is given.
    E.4Principal exclusion criteria
    - Chronic blood transfusion or simple transfusion in the preceding 3 months
    - Painful crises in the past 4 weeks
    - Pregnancy, breast feeding or desire to get pregnant in the following 7 months
    - Known active gastric ulcers
    - Hydroxycarbamide treatment with unstable dose in the past 3 months or Hydroxycarbamide commenced less than 6 months prior to study
    - Insufficient compliance in the run in period (Participants that do not show up for their follow-up visit without prior notice, that not bring
    their diary or with less than 80% of the days in the pain diary filled in, will be excluded from participation in this trial and will not be randomized.)
    - Known poor compliance in earlier trials regarding the completion of pain diaries.
    - Known hypersensitivity to N-acetylcysteine or one of its components.
    - Use of pain medication for sickle-cell related pains on more than 15 days per month
    in the past 6 months (‘chronic pain’).
    E.5 End points
    E.5.1Primary end point(s)
    The main endpoint of this study is the frequency of SCD related pain in daily life in patients with SCD. Frequency of pain will be expressed in days per patient in proportion to the total time of intervention (6 months) per treatment group.
    E.5.1.1Timepoint(s) of evaluation of this end point
    By use of a daily pain diary (monthly evaluated during 6 months)
    E.5.2Secondary end point(s)
    - The severity of sickle cell related pain in daily life
    - The frequency and severity of painful crises in daily life
    - The frequency and length of hospital admission for painful crises
    - Time to first and second painful crisis and hospital admission for painful crisis
    - Health related QoL
    - Societal costs of SCD related pain care
    - Hematological markers of oxidative stress, hemolysis, hypercoagulability, inflammation and endothelial dysfunction.
    - Tolerability of NAC
    - Frequency of use of pain medication at home
    - Related incidence of SCD complications (e.g. acute chest syndrome)
    E.5.2.1Timepoint(s) of evaluation of this end point
    baseline, 3 months and 6 months
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety No
    E.6.5Efficacy No
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA10
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days1
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months1
    E.8.9.2In all countries concerned by the trial days1
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 40
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 40
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 100
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state40
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 140
    F.4.2.2In the whole clinical trial 140
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Academic Medical Center, Department of Haematology, University of Amsterdam
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2015-01-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-01-30
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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